Recent advances in immune modulation

Current Gene Therapy

Volumn 7, Issue 5, 2007, Pages 391-402

  Miao, Carol H ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Gene therapy; Gene transfer; Genetic diseases; Hemophilia; Immune responses; Immunomodulation; Immunosuppression; Inhibitory antibody

Indexed keywords

ABATACEPT; ALEMTUZUMAB; ANTIBODY; BUSULFAN; CD20 ANTIBODY; CD3 ANTIBODY; CD40 LIGAND MONOCLONAL ANTIBODY; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; DACLIZUMAB; GENE PRODUCT; IMMUNOSUPPRESSIVE AGENT; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; OKT 3; RAPAMYCIN; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; RITUXIMAB; TACROLIMUS; THYMOCYTE ANTIBODY;

ANTIBODY PRODUCTION; ANTIGEN EXPRESSION; APOPTOSIS; CLINICAL PROTOCOL; CRIGLER NAJJAR SYNDROME; CYTOTOXICITY; DRUG TARGETING; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GRAFT REJECTION; GRAFT SURVIVAL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOPHILIA A; HEMOPHILIA B; HUMAN; IMMUNE RESPONSE; IMMUNOLOGICAL TOLERANCE; IMMUNOMODULATION; LYMPHATIC LEUKEMIA; LYMPHOCYTE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; ORGAN TRANSPLANTATION; REGULATORY T LYMPHOCYTE; REVIEW; T LYMPHOCYTE; T LYMPHOCYTE ACTIVATION; THYMUS; TRANSGENE;

ANIMALS; ANTIBODIES, MONOCLONAL; ANTIGENS, CD; APOPTOSIS; AUTOIMMUNE DISEASES; CLONAL ANERGY; CLONAL DELETION; GENE THERAPY; HUMANS; IMMUNE TOLERANCE; IMMUNOSUPPRESSION; IMMUNOSUPPRESSIVE AGENTS; LYMPHOCYTE ACTIVATION; MODELS, IMMUNOLOGICAL; T-LYMPHOCYTES, HELPER-INDUCER; T-LYMPHOCYTES, REGULATORY; TRANSGENES; TRANSPLANTATION IMMUNOLOGY; TRANSPLANTATION TOLERANCE;

EID: 35349031236     PISSN: 15665232     EISSN: None     Source Type: Journal    
DOI: 10.2174/156652307782151524     Document Type: Review

References (143)

1. Abrams, J.R., Lebwohl, M.G., Guzzo, C.A., Jegasothy, B.V., Goldfarb, M.T., Goffe, B.S., Menter, A., Lowe, N.J., Krueger, G., Brown, M.J., Weiner, R.S., Birkhofer, M.J., Warner, G.L., Berry, K.K., Linsley, P.S., Krueger, J.G., Ochs, H.D., Kelley, S.L. and Kang, S. (1999). CTLA4Ig-mediated blockade of T-cell costimulation in patients with psoriasis vulgaris. J. Clin. Invest., 103: 1243-52.
2. Adams, A.B., Shirasugi, N., Jones, T.R., Durham, M.M., Strobert, E.A., Cowan, S., Rees, P., Hendrix, R., Price, K., Kenyon, N.S., Hagerty, D., Townsend, R., Hollenbaugh, D., Pearson, T.C. and Larsen, C.P. (2005). Development of a chimeric anti-CD40 monoclonal antibody that synergizes with LEA29Y to prolong islet allograft survival. J. Immunol., 174: 542-50.
3. Aggarwal, A., Grewal, R., Green, R.J., Boggio, L., Green, D., Weksler, B.B., Wiestner, A. and Schechter, G.P. (2005). Rituximab for autoimmune haemophilia: a proposed treatment algorithm. Haemophilia, 11: 13-9.
4. Andrews, J.L., Kadan, M.J., Gorziglia, M.I., Kaleko, M. and Connelly, S. (2001). Generation and characterization of El/E2a/E3/E4-deficient adenoviral vectors encoding human factor VIII. Mol. Ther., 3: 329-36.
5. Arruda, V.R., Stedman, H.H., Nichols, T.C., Haskins, M.E., Nicholson, M., Herzog, R.W., Couto, L.B. and High, K.A. (2005). Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood, 105: 3458-64.
6. Behara, A.M., Westcott, A.J. and Chang, P.L. (1992). Intrathymic implants of genetically modified fibroblasts. FASEB J., 6: 2853-8.
7. Beier, K.C., Hutloff, A., Dittrich, A.M., Heuck, C., Rauch, A., Buchner, K., Ludewig, B., Ochs, H.D., Mages, H.W. and Kroczek, R.A. (2000). Induction, binding specificity and function of human ICOS. Eur. J. Immunol., 30: 3707-17.
8. Berntorp, E., Astermark, J. and Carlborg, E. (2000). Immune tolerance induction and the treatment of hemophilia. Malmo protocol update. Haematologica, 85: 48-50; discussion 50-1.
9. Beutelspacher, S.C., Pillai, R., Watson, M.P., Tan, P.H., Tsang, J., McClure, M.O., George, AJ. and Larkin, D.F. (2006). Function of indoleamine 2,3-dioxygenase in corneal allograft rejection and prolongation of allograft survival by over-expression. Eur. J. Immunol., 36: 690-700.
10. Billingham, R.E., Brent; L. and Medawar, P.B. (1953). Actively acquired tolerance of foreign cells. Nature, 172: 603-6.
11. Biss, T.T., Velangi, M.R. and Hanley, J.P. (2006). Failure of rituximab to induce immune tolerance in a boy with severe haemophilia A and an alloimmune factor VIII antibody: a case report and review of the literature. Haemophilia, 12: 280-4.
12. Blaha, P., Bigenzahn, S., Koporc, Z., Sykes, M., Muechlbacher, F. and Wekerle, T. (2005). Short-term immunosuppression facilitates induction of mixed chimerism and tolerance after bone marrow transplantation without cytoreductive conditioning. Transplantation, 8: 237-43.
13. Bresson, D., Togher, L., Rodrigo, E., Chen, Y., Bluestone, J.A., Herold, K.C. and von Herrath, M. (2006). Ami-CD3 and nasal proinsulin combination therapy enhances remission from recent-onset autoimmune diabetes by inducing Tregs. J. Clin. Invest., 116: 1371-81.
14. Briet, E, and Mauser-Bunschoten, E.P. (1977). Revision consensus hemophilia: treatment and responsibility. Nederlandse Vereniging van Hemophilia Patients. Ned. Tijdschr Geneeskd, 141: 2566-71.
15. Cao, O., Armstrong, E., Schlachterman, A., Wang, L., Okita, D.K., Conti-Fine, B., High, K.A. and Herzog, R.W. (2006). Immune deviation by mucosal antigen administration suppresses gene-transfer-induced inhibitor formation to factor DC. Blood, 108: 480-6.
16. Cao, O.W., Dobrzynski, E., Wang, L., Nayak, S., Mingle, B., Terhorst, C.P. and Herzog, R.W. (2007). Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood, 110: 1132-40.
17. Carlborg, E., Astermark, J., Lethagen, S., Ljung, R. and Berntorp, E. (2000). The Malmo model for immune tolerance induction: impact of previous treatment on outcome. Haemophilia, 6: 639-42.
18. Chao, H., Mao, L., Bruce, A.T. and Walsh, C.E. (2000). Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood, 95: 1594-9.
19. Coenen, J.J., Koenen, H.J., van Rijssen, E., Hilbrands, L.B. and Joosten, I. (2005). Tolerizing effects of co-stimulation blockade rest on functional dominance of CD4+CD25+ regulatory T cells. Transplantation, 79: 147-56.
20. Coiffier, B., Lepage, E., Briere, J., Herbrecht, R., Tilly, H., Bouabdallah, R., Morel, P., Van Den Neste, E., Salles, G., Gaulard, P., Reyes, F., Lederlin, P. and Gisselbrecht, C. (2002). CHOP chemotherapy plus rituximab compared with CHOP alone in elderly patients with diffuse large-B-cell lymphoma. N Engl. J. Med., 346: 235-42.
21. Dai, Y., Schwarz, E.M., Gu, D., Zhang, W.W., Sarvetnick, N. and Verma, I.M. (1995). Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl. Acad. Sci. USA, 92: 1401-5.
22. Dall'Era, M. and Davis, J. (2004). CTLA4Ig: a novel inhibitor of costimulation. Lupus, 13: 372-6.
23. DeMatteo, R.P., Raper, S.E., Ahn, M., Fisher, K.J., Burke, C., Radu, A., Widera; G., Claytor, B.R., Barker, C.F. and Markmann, J.F. (1995). Gene transfer to the thymus. A means of abrogating the immune response to recombinant adenovirus. Ann. Surg., 222: 229-39; discussion 239-42.
24. Doering, C.B., Gangadharan, B., Dukart, H.Z. and Spencer, H.T. (2007). Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity. Mol. Ther., 15: 1093-9.
25. Edinger, M., Hoffmann, P., Ermann, J., Drago, K., Fathman, C.G, Strober, S. and Negrin, R.S. (2003). CD4+CD25+ regulatory T cells preserve graft-versus-tumor activity while inhibiting graft-versus-host disease after bone marrow transplantation. Nat. Med., 9: 1144-50.
26. Evans, G.L. and Morgan, R.A. (1998). Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc. Natl. Acad. Sci. USA, 95: 5734-9.
27. Fandrich F., Zhu, X., Schroder, J., Dresske, B., Henne-Bruns, D., Oswald, H. and Zavazava, N. (1999). Different in vivo tolerogenicity of MHC class I peptides. J. Leukoc. Biol., 65: 16-27.
28. Fang, B., Eisensmith, R.C., Wang, H., Kay, M.A., Cross, R.E., Landen, C.N., Gordon, G., Bellinger, D.A., Read, M.S., Hu, P.C. and Brinkhous, K.M. and Woo, S.L.C. (1995). Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Hum. Gene. Ther., 6: 1039-44.
29. Fields, P.A., Arruda, V.R., Armstrong, E., Chu, K., Mingozzi, F., Hagstrom, J.N., Herzog, R.W. and High, K.A. (2001). Risk and prevention of antifactor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. Mol. Ther., 4: 201-10.
30. Fields, P.A., Kowalczyk, D.W., Arruda, V.R., Armstrong, E., McCleland, M.L., Hagstrom, J.N., Pasi, K.J., Ertl, H.C., Herzog, R.W. and High, K.A. (2000). Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. Mol. Ther., 1: 225-35.
31. Fontenot, J.D., Gavin, M.A. and Rudensky, A.Y. (2003). Foxp3 programs the development and function of CD4+CD25+ regulatory T cells. Nat. Immunol., 4: 330-6.
32. Fox, R.A., Neufeld, E.J. and Bennett, C.M. (2006). Rituximab for adolescents with haemophilia and high titre inhibitors. Haemophilia, 12: 218-22.
33. Foy, T., Shepherd, D. and Durle, F.E.A. (1993). In vivo CD40-gp39 onteractions are essential for thymus dependent humoral immunity, II: prolonged suppression of the humoral immune response by an antibody to the ligand for CD40, gp39. J. Exp. Med., 178: 1567-1575.
34. Friend, P.J., Hale, G., Chatenoud, L., Rebello, P., Bradley, J., Thiru, S., Phillips, J.M. and Waldmann, H. (1999). Phase I study of an engineered aglycosylated humanized CD3 antibody in renal transplant rejection. Transplantation, 68: 1632-7.
35. Golshayan, D., Buhler, L., Lechler, R.I. and Pascual, M. (2007). From current immunosuppressive strategies to clinical tolerance of allografts. Transpl. Int., 20: 12-24.
36. Grabowski, G.A., Barton, N.W., Pastores, G., Dambrosia, J.M., Banerjee, T.K., McKee, M.A., Parker, C., Schiffmann, R., Hill, S.C. and Brady, R.O. (1995). Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann. Intern. Med., 122: 33-9.
37. Greengard, J.S. and Jolly, P.J. (1999). Animal testing of retroviral-mediated gene therapy for factor VIII deficiency. Thromb. Haemost., 82: 555-61.
38. Guillonneau, C., Aubry, V., Renaudin, K., Seveno, C., Usal, C., Tezuka, K. and Anegon, I. (2005). Inhibition of chronic rejection and development of tolerogenic T cells after ICOS-ICOSL and CD40-CD40L co-stimulation blockade. Transplantation, 80: 546-54.
39. Guillonneau, C., Hill, M., Hubert, F.X., Chiffaleau, E., Herve, C., Li, X.L., Heslan, M., Usal, C., Tesson, L., Menoret, S., Saoudi, A., Le Mauff, B., Josien, R., Cuturi, M.C. and Anegon, I. (2007). CD40Ig treatment results in allograft acceptance mediated by CD8CD45RC T cells, IFN-gamma, and indoleamine 2,3-dioxygenase. J. Clin. Invest., 117: 1096-106.
40. Guillot, C., Guillonneau, C., Mathieu, P., Gerdes, C.A., Menoret, S., Braudean, C., Tesson, L., Renaudin, K., Castro, M.G., Lowenstein, P.R. and Anegon, I. (2002). Prolonged blockade of CD40-CD40 ligand interactions by gene transfer of CD40Ig results in long-term heart allograft survival and donor-specific hyporesponsiveness, but does not prevent chronic rejection. J. Immunol., 168: 1600-9.
41. Harmeling, B.R., Ziegler, S., Torgerson, T., Chen, L., Ochs, H.D., Rawlings, D.J. and Miao, C.H. (2007). Regulation of FVIII-specific immune responses in a plasmid DNA treated hemoṕhilia A mouse model by transgenic FOXP3+CD4+ regulatory T cells. Mol. Ther., 15, Suppl. 1: S97.
42. Hausl, C., Ahmad, R.U., Sasgary, M., Doering, C.B., Lollar, P., Richter, G., Schwarz, H.P., Turecek, P.L. and Reipert, B.M. (2005). High-dose factor VIII inhibits factor VIII-specific memory B cells in hemophilia A with factor VIII inhibitors. Blood, 106: 3415-22.
43. Hedner, U. and Kisiel, W. (1983). Use of human factor VIIa in the treatment of two hemophilia A patients with high-titer inhibitors. J. Clin. Invest., 71: 1836-41.
44. Hering, B.J., Kandaswamy, R., Harmon, J.V., Ansite, J.D., Clemmings, S.M., Sakai, T., Paraskevas, S., Eckman, P.M., Sageshima, J., Nakano, M., Sawada, T., Matsumoto, I., Zhang, H.J., Sutherland, D.E. and Bluestone, J.A., (2004). Transplantation of cultured islets from two-layer preserved pancreases in type 1 diabetes with anti-CD3 antibody. Am. J. Transplant., 4: 390-401.
45. Herold, K.C., Hagopian, W., Auger, J.A., Poumian-Ruiz, E., Taylor, L., Donaldson, D., Gitelman, S.E., Harlan, D.M., Xu, D., Zivin, R.A. and Bluestone, J.A. (2002). Anti-CD3 monoclonal antibody in new-onset type 1 diabetes mellitus. N. Engl. J. Med., 346: 1692-8.
46. Herzog, R.W. and Dobrzynski, E. (2004). Immune implications of gene therapy for hemophilia. Semin. Thromb. Hemost., 30: 215-26.
47. Herzog, R.W., Fields, P.A., Arruda, V.R., Brubaker, J.O., Armstrong, E., McClintock D., Bellinger, D.A., Couto, L.B., Nichols, T.C. and High, K.A. (2002). Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum. Gene. Ther., 13: 1281-91.
48. Herzog, R.W., Mount, J.D., Arruda, V.R., High, K.A. and Lothrop, C.D., Jr. (2001). Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Mol. Ther., 4: 192-200.
49. Hoffmann, P., Ermann, J., Edinger, M., Fathman, C.G. and Strober, S. (2002). Donor-type CD4(+)CD25(+) regulatory T cells suppress lethal acute graft-versus-host disease after allogeneic bone marrow transplantation. J. Exp. Med., 196: 389-99.
50. Homan, D., Dyrberg, T., Petersen, J., Oldstone, M.B. and von Herrath, M.G. (1999). Insulin in oral immune "tolerance": a one-amino acid change in the B chain makes the difference. J. Immunol., 163: 1833-8.
51. Hori, S., Nomura, T. and Sakaguchi, S. (2003). Control of regulatory T cell development by the transcription factor Foxp3. Science, 299: 1057-61.
52. Hoyer, L.W. and Scandella, D. (1994). Factor VIII inhibitors: structure and function in autoantibody and hemophilia A patients. Semin. Hematol., 31: 1-5.
53. Ide, L.M., Gangadharan, B., Chiang, K.Y., Doering, C.B. and Spencer, H.T. (2007). Hematopoietic stem cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood, June 25, Epub ahead of print.
54. Ilan, Y., Attavar, P., Takahashi, M., Davidson, A., Horwitz, M.S., Guida, J., Chowdhury, N.R. and Chowdhury, J.R. (1996). Induction of central tolerance by intrathymic inoculation of adenoviral antigens into the host thymus permits long-term gene therapy in Gunn rats. J. Clin. Invest., 98: 2640-7.
55. Ilan, Y., Jona, V.K., Sengupta, K., Davidson, A., Horwitz, M.S., Roy-Chowdhury, N. and Roy-Chowdhury, J. (1997). transient immunosupression with FK506 permits long-term expression of therapeutic genes introduced into the liver using recombinant adenoviruses in the rat. Hepatology, 26: 949-56.
56. Jacquemin, M., Vantomme, V., Buhot, C., Lavend'homme, R.; Burhy, W., Demotte, N., Chaux, P., Peerlinck, K., Vermylen, J., Maillere, B., van der Bruggen, P. and Saint-Remy, J.M. (2003). CD4+ T-cell clones specific for wild-type factor VIII: a molecular mechanism responsible for a higher incidence of inhibitor formation in mild/moderate hemophilia A. Blood, 101: 1351-8.
57. Jaeckel, E., von Boehmer, H. and Manns, M.P. (2004). Antigen-Specific FoxP3-Transduced T-Cells Can Control Established Type 1 Diabetes. Diabetes, 54: 306-10.
58. Jiang, H., Couto, L.B., Patarroyo-White, S., Liu, T., Nagy, D., Vargas, J.A. Zhou, S., Scallan, C.D., Sommer, J., Vijay, S., Mingozzi, F., High, K.A. and Pierce, G.F. (2006). Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macajues and implications for human gene therapy. Blood, 108: 3321-8.
59. Jiang, Z., Schiedner, G., van Rooijen, N., Liu, C.C., Kochanek, S. and Clemens, P.R. (2004). Sustained muscle expression of dystrophin from a high-capacity adenoviral vector with systemic gene transfer of T cell costimulatory blockade. Mol. Ther., 10: 688-96.
60. Jiang, Z.L., Reay, D., Kreppel, F., dambotto, A., Feingold, E., Kochanek, S., McCarthy, S.A. and Clemens, P.R. (2001). Local high-capacity adenovirus-mediated mCTLA4Ig and mCD40Ig expression prolongs recombinant gene expression in skeletal muscle. Mol. Ther., 3: 892-900.
61. Kaihara, S., Bessho, K., Okubo, Y., Sonobe, T., Kawai, M. and Iizuka, T. (2004). Simple and effective osteoinductive gene therapy by local injection of a bone morphogenetic protein-2-expressing recombinant adenoviral vector and FK506 mixture in rats. Gene Ther. 11: 439-47.
62. Kasprowicz, D.J., Smallwood, P.S., Tyznik, A.J. and Ziegler, S.F. (2003). Scurfin (FoxP3) controls T-dependent immune responses in vivo through regulation of CD4+ T cell effector function. J. Immunol., 171: 1216-23.
63. Kay, M.A., Landen, C.N., Rothenberg, S.R., Taylor, L.A., Leland, F., Wiehle, S., Fang, B., Bellinger, D., Finegold, M., Thompson, A.R. (1994). "In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc. Natl. Acad. Sci. USA, 91: 2353-7.
64. Kay, M.A., Meuse, L., Gown, A.M., Linsley, P., Hollenbaugh, D., Aruffo, A., Ochs, H.D. and Wilson, C.B. (1997). Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver. Proc. Natl. Acad. Sci. USA, 94: 4686-91.
65. Khattri, R., Cox, T., Yasayko, S.A. and Ramsdell, F. (2003). An essential role for Scurfin in CD4+CD25+ T regulatory cells. Nat. Immunol. 4: 337-42.
66. Larsen, C.P., Elwood, E.T., Alexander, D.Z., Ritchie, S.C., Hendrix, R., Tucker-Burden, C., Cho, H.R., Aruffo, A., Hollenbaugh, D., Linsley, P.S., Winn, K.J. and Pearson, T.C. (1996). Long-term acceptance of skin and cardiac allografts after blocking CD40 and CD28 pathways. Nature, 381: 434-8.
67. Larsen, C.P., Pearson, T.C., Adams, A.B., Tso, P., Shirasugi, N., Strobertm, E., Anderson, D., Cowan, S., Price, K., Naemura, J., Emswiler, J., Greene, J., Turk, L.A., Bajorath, J., Townsend, R., Hagerty, D., Linsley, P.S. and Peach, R.J. (2005). Rational development of LEA29Y (belatacept), a high-affinity variant of CTLA4-Ig with potent immunosuppressive properties. Am. J. Transplant., 5: 443-53.
68. Lei, T.C., Su, Y. and Scott, D.W. (2005). Tolerance induction via a B-cell delivered gene therapy-based protocol: optimization and role of the Ig scaffold. Cell Immunol., 235: 12-20.
69. Liu, H., Liu, L., Fletcher, B.S. and Visner, G.A. (2006). Sleeping Beauty-based gene therapy with indoleamine 2,3-dioxygenase inhibits lung allograft fibrosis. FASEB J., 20: 2384-6.
70. Lundquist, L. (2007). Abatacept: a novel therapy approved for the treatment of patients with rheumatoid arthritis. Adv. Ther., 24: 333-45.
71. Lusher, J.M. (2000). Inhibitor antibodies to factor VIII and factor IX: management. Semin. Thromb. Hemost. 26: 179-88.
72. Ma, X., Liu, Y., Tittiger, M., Hennig, A., Kovacs, A., Popelka, S., Wang, B., Herati, R., Bigg, M. and Ponder, K.P. (2007). Improvements in Mucopolysaccharidosis I Mice After Adult Retroviral Vector-mediated Gene Therapy with Immunomodulation. Mol. Ther., 15: 889-902.
73. Magliocca, J.F. and Knechtle, S.J. (2006). The evolving role of alemtuzumab (Campath-1H) for immunosuppressive therapy in organ transplantation. Transpl. Int., 19: 705-14.
74. Marodon, G., Fisson, S., Levacher, B., Fabre, M., Salomon, B.L. and Klatzmann, D. (2006). Induction of antigen-specific tolerance by intrathymic injection of lentiviral vectors. Blood, 108: 2972-8.
75. Mellor, A.L., Baban, B., Chandler, P., Marshall, B., Jhaver, K., Hansen, A., Koni, P.A., Iwashima, M. and Munn, D.H. (2003). Cutting edge: induced indoleamine 2,3 dioxygenase expression in dendritic cell subsets suppresses T cell clonal expansion. J. Immunol., 171: 1652-5.
76. Mellor, A.L., Chandler, P., Baban, B., Hansen, A.M., Marshall, B., Pihkala, J., Waldmann, H., Cobbold, S., Adams, E. and Munn, D.H. (2004). Specific subsets of murine dendritic cells acquire potent T cell regulatory functions following CTLA4-mediated induction of indoleamine 2,3 dioxygenase. Int. Immunol., 16: 1391-401.
77. Miao, C.H., Ye, P., Thompson, A.R., Rawlings, D.J. and Ochs, H.D. (2006). Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice. Blood, 108: 19-27.
78. Mao, C.H., Ye, X. and Thompson, A.R. (2003). High-level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors. Hum. Gene Ther., 14: 1297-305.
79. Mingozzi, F., Hasbrouck, N.C., Basner-Tschkarajan, E., Edmonson, S.A., Hui, D.J., Sabatino, D.E., Zhou, S., Wright, J.F., Jiang, H., Pierce, G.F., Arruda, V.R. and High, K.A. (2007). Modulation of tolerance to the transgene product in a non-human primate model of AAV-mediated gene transfer to liver. Blood, Jul 3: [Epub ahead of print].
80. Mingozzi, F., Liu, Y.L., Dobrzynski, E., Kaufhold, A., Liu, J.H., Wang, Y., Arruda, V.R., High, K.A. and Herzog, R.W. (2003). Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J. Clin. Invest., 111: 1347-56.
81. Moayeri, M., Hawley, T.S. and Hawley, R.G. (2005). Correction of murine hemophilia A by hematopoietic stem cell gene therapy. Mol. Ther., 12: 1034-42.
82. Nanji, S.A., Hancock, W.W., Luo, B., Schur, C.D., Pawlick, R.L., Zhu, L.F., Anderson, C.C. and Shapiro, A.M. (2006). Costimulation blockade of both inducible costimulator and CD40 ligand induces dominant tolerance to islet allografts and prevents spontaneous autoimmune diabetes in the NOD mouse. Diabetes, 55: 27-33.
83. Nathwani, A.C., Gray, J.T., Ng, C.Y., Zhou, J., Spence, Y., Waddington, S.N., Tuddenham, E.G., Kemball-Cook, G., McIntosh, J., Boon-Spijker, M., Mertens, K. and Davidoff, A.M. (2006). Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood, 107: 2653-61.
84. Nicolls, M.R., Aversa, G.G. Pearce, N.W., Spinelli, A., Berger, M.F., Gurley, K.E. and Hall, B.M. (1993). Induction of long-term specific tolerance to allografts in rats by therapy with an anti-CD3-like monoclonal antibody. Transplantation, 55: 459-68.
85. Ochando, J.C., Yopp, A.C., Yang, Y., Garin, A., Li, Y., Boros, P., Llodra, J., Ding, Y., Lira, S.A., Krieger, N.R. and Bromberg, J.S. (2005). Lymph node occupancy is required for the peripheral development of alloantigen-specific Foxp3+ regulatory T cells. J. Immunol., 174: 6993-7005.
86. Ohyama, M., Ota, T., Aoki, M., Tsunoda, K., Harada, R., Koyasu, S., Nishikawa, T. and Amagai, M. (2003). Suppression of the immune response against exogenous desmoglein 3 in desmoglein 3 knockout mice: an implication for gene therapy. J. Invest. Dermatol., 120: 610-5.
87. Oluwole, S.F., Chowdhury, N.C., Ingram, M., Garrovillo, M., Jin, M.X. and Agrawal, S. (1999). Mechanism of acquired thymic tolerance induced by a single major histocompatibility complex class I peptide with the dominant epitope: differential analysis of regulatory cytokines in the lymphoid and intragraft compartments. Transplantation, 68: 418-29.
88. Peng, B., Ye, P., Blazer, B.R., Ochs, H.D. and Miao, C.H. (2007). Transient blockade of ICOS co-stimulatory pathways induces long-term tolerance to Factor VIII following nonviral gene transfer of Factor VIII plasmid into hemophilia A mice. Mol. Ther., 15, Suppl. 1: S415.
89. Petrof, B.J., Acsadi, G., Jani, A., Massie, B., Bourdon, J., Matusiewicz, N., Yang, L., Lochmuller, H. and Karpati, G. (1995). Efficiency and functional consequences of adenovirus-mediated in vivo gene transfer to normal and dystrophic (mdx) mouse diaphragm. Am. J. Respir. Cell Mol. biol., 13: 508-17.
90. Plain, K.M., Chen, J., Merten, S., He, X.Y. and Hall, B.M. (1999). Induction of specific tolerance to allografts in rats by therapy with non-mitogenic, non-depleting anti-CD3 monoclonal antibody: association with TH2 cytokines not anergy. Transplantation, 67: 605-13.
91. Potter, M.A., Hymus, S., Stockley, T. and Chang, P.L. (1998). Suppression of immunological response against a transgene product delivered from microencapsulated cells. Hum. Gene Ther., 9: 1275-82.
92. Puppi, J., Guillonneau, C., Pichard, V., Bellodi-Privato, M., Cuturi, M.C., Anegon, I. and Ferry, N. (2004). Long term transgene expression by hepatocytes transduced with retroviral vectors requires induction of immune tolerance to the transgene. J. Hepatol., 41: 222-8.
93. Qian, J., Collins, M., Sharpe, A.H. and Hoyer, L.W. (2000). Prevention and treatment of factor VIII inhibitors in murine hemophilia A. Blood 95: 1324-9.
94. Quezada, S.A., Jarvinen, L.Z., Lind, E.F. and Noelle, R.J. (2004). CD40/ CD154 interactions at the interface of tolerance and immunity. Annu. Rev. Immunol., 22: 307-28.
95. Rawle, F.E., Pratt, K.P., Labelle, A., Weiner, H.L., Hough, C. and Lillicrap, D. (2006). Induction of partial immune tolerance to factor VIII through prior mucosal exposure to the factor VIII C2 domain. J. Thromb. Haemost., 4: 2172-9.
96. Rawle, F.E.M., Labelle, A.D., Davie, E., Pratt, K. and Lillicrap, D.P. (2004). Prevention of anti-FVIII inhibitor formation post protein infusion in hemophilic mice by prior feeding of the FVIII-C2 domain. Blood, 104: 15A.
97. Read, S., Malmstrom, V. and Powrie, F. (2000). Cytotoxic T lymphocyte-associated antigen 4 plays an essential role in the function of CD25(+)CD4(+) regulatory cells that control intestinal inflammation. J. Exp. Med., 192: 295-302.
98. Richards, S.M., Olson, T.A. and McPherson, J.M. (1993). Antibody response in patients with Gaucher disease after repeated infusion with macrophage-targeted glucocerebrosidase. Blood, 82: 1402-9.
99. Roberts H.R. and Eberst, M.E. (1993). Current management of hemophilia B. Hematol. Oncol. Clin. North Am., 7: 1269-80.
100. Ross, C.J., Twisk, J., Bakker, A.C., Miao, F., Verbart, D., Rip, J., Godbey, T., Dijkhuizen, P., Hermens, W.T., Kastelein, J.J., Kuivenhoven, J.A., Meulenberg, J.M. and Hayden, M.R. (2006). Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum. Gene Ther., 17: 487-99.
101. Rossi, G., Sarkar, J. and Scandella, D. (2001). Long-term induction of immune tolerance after blockade of CD40-CD40L interaction in a mouse model of hemophilia A. Blood, 97: 2150-7.
102. Roy, K., Mao, H.Q., Huang, S.K. and Leong, K.W. (1999). Oral gene delivery with chitosan - DNA nanoparticles generates immunologic protection in amurine model of peanut allergy. Nat. Med., 5: 387-91.
103. Saenko, E.L., Ananyeva, N.M., Moayeri, M., Ramezani, A. and Hawley, R.G. (2003). Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A. Curr. Gene Ther., 3: 27-41.
104. Sakaguchi, S. (2000). Regulatory T cells: key controllers of immunologic self-tolerance. Cell, 101: 455-8.
105. Salomon, B., Lenschow, D.J., Rhee, L., Ashourian, N., Singh, B., Sharpe, A. and Bluestone, J.A. (2000). B7/CD28 costimulation is essential for the homeostasis of the CD4+CD25+ immunoregulatory T cells that control autoimmune diabetes. Immunity, 12: 431-40.
106. Sampaolesi, M., Blot, S., D'Antona, G., Granger, N., Tonlorenzi, R., Innocenzi, A., Mognol, P., Thibaud, J.L., Galvez, B.G., Barthelemy, I., Perani, L., Mantero, S., Guttinger, M., Pansarasa, O., Rinaldi, C., Cusella De Angelis, M.G., Torrente, Y., Bordignon, C., Bottinelli, R. and Cossu, G. (2006). Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature, 444: 574-9.
107. Sands, M.S., Vogler, C., Kyle, J.W., Grubb, J.H., Levy, B., Galvin, N., Sly, W.S. and Birkenmeier, E.H. (1994). Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J. Clin. Invest., 93: 2324-31.
108. Sarkar, R., Gao, G.P., Chirmule, N., Tazelaar, J. and Kazazian, H.H.J. (2000). Partial corretion of murine hemophilia A with neo-antigenic murine factor VIII,. Hum. Gene. Ther., 11: 881-94.
109. Sarkar, R., Mucci, M., Addya, S., Tetreault, R., Bellinger, D.A., Nichols, T.C. and Kazazian, H.H., Jr. (2006). Long-term efficacy of adeno-associated virus serotys 8 and 9 in hemophilia a dogs and mice. Hum. Gene Ther., 17: 427-39.
110. Schowalter, D.B., Meuse, L., Wilson, C.B., Linsley, P.S. and Kay, M.A. (1997). Constitutive expression of murine CTLA4Ig from a recombinant adenovirus vector results in prolonged transgene expression. Gene Ther., 4: 853-60.
111. Scott, D. (1994). The Nature of Immunologic Tolerance. Austin USA: RG Landes Co. 1994.
112. Shapiro, A. (2003). Inhibitor treatment: state of the art. Dis. Mon., 49: 22-38.
113. Shevach, E.M. (2002). CD4+ CD25+ suppressor T cells: more questions than answers. Nat. Rev. Immunol., 2: 389-400.
114. Shull, R., Lu, X., Dube, I., Lutzko, C., Kruth, S., Abrams-Ogg, A., Kiem, H.P., Goehle, S., Schuening, F., Millan, C. and Carter, R. (1996). Humoral immune response limits gene therapy in canine MPS I. Blood, 88: 377-9.
115. Smakman, N., van der Bilt, J.D., van den Wollenberg, D.J., Hoeben, R.C., Borel Rinkes, I.H. and Kranenburg, O. (2006). Immunosuppression promotes reovirus therapy of colorectal liver metastases. Cancer Gene Ther., 13: 815-8.
116. Smith, R. (1961). Immunologic tolerance in non-living antignes. Adv. Immunol., 1: 67.
117. Stein, C.S., Kang, Y., Sauter, S.L., Townsend, K., Staber, P., Derksen, T.A., Martins, I., Qian, J., Davidson, B.L. and McCray, P.B., Jr. (2001). In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. Mol. Ther., 3: 850-6.
118. Swanson, K.A., Zheng, Y., Heidler, K.M., Mizobuchi, T. and Wilkes, D.S. (2004). CD11c+ cells modulate pulmonary immune responses by production of indoleamine 2,3-dioxygenase. Am. J. Respir. Cell Mol. Biol. 30: 311-8.
119. Taylor, P.A., Lees, C.J. and Blazar, B.R. (2002). The infusion of ex vivo activated and expanded CD4(+)CD25(+) immune regulatory cells inhibits graft-versus-host disease lethality. Blood, 99: 3493-9.
120. Taylor, P.A., Panoskaltsis-Mortari, A., Freeman, G.J., Sharpe, A.H., Noelle, R.J., Rudensky, A.Y., Mak, T.W., Serody, J.S. and Blazar, B.R. (2005). Targeting of inducible costimulator (ICOS) expressed on alloreactive T cells down-regulates graft-versus-host disease (GVHD) and facilitates engraftment of allogeneic bone marrow (BM). Blood, 105: 3372-80.
121. Thummala, N.R., Ghosh, S.S., Lee, S.W., Reddy, B., Davidson, A., Horwitz, M.S., Chowdhury, J.R. and Chowdhury, N.R. (2002). A non-immunogenic adenoviral vector, coexpressing CTLA4Ig and bilirubinuridine-diphosphoglucuronateglucuronosyltransferase permits long-term, repeatable transgene expression in the Gunn rat model of Crigler-Najjar syndrome. Gene Ther., 9: 981-90.
122. Toromanoff, A., Hill, M., Cherel, Y., Deschamps, J.-Y., Gauthier, O., Vanhove, B., Rolling, F., Moulier, P., Anegon, I. and Guiner, C.L. (2006). "Constitutive Expression of a Mutated Form of CTLA4Ig after Gene Transfer Is Well Tolerated and Prevents Immune Responses Against an Immunogenic Transgene in Nonhuman Primates." Mo.l Ther., 13: S375.
123. Utset, T.O., Auger, J.A., Peace, D., Zivin, R.A., Xu, D., Jolliffe, L., Alegre, M.L., Bluestone, J.A. and Clark, M.R. (2002). Modified anti-CD3 therapy in psoriatic arthritis: a phase I/II clinical trial. J. Rheumatol., 29: 1907-13.
124. Van Cott, K.E., Monahan, P.E., Nichols, T.C. and Velander, W.H. (2004). Haemophilic factors produced by transgenic livestock: abundance that can enable alternative therapies worldwide. Haemophilia, 10 Suppl 4: 70-6.
125. VandenDriessche, T., Collen, D. and Chuah, M.K. (2003). Gene therapy for the hemophilias. J. Thromb. Haemost., 1: 1550-8.
126. VandenDriessche, T., Vanslembrouck, V., Goovaerts, I., Zwinnen, H., Vanderhaeghen, M.L., Collen, D. and Chuah, M.K. (1999). Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc. Natl. Acad. Sci. USA, 96:0379-84.
127. Vanhove, B. and Soulillou, J.P. (2005). Technology evaluation: Belatacept, Bristol-Myers Squibb. Curr. Opin. Mol. Ther., 7: 384-93.
128. Waddington, S.N., Buckley, S.M., Nivsarkar, M., Jezzard, S., Schneider, H., Dahse, T., Kemball-Cook, G., Miah, M., Tucker, N., Dallman, M.J., Themis, M. and Coutelle, C. (2003). In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. Blood, 101: 1359-66.
129. Waddington, S.N., Nivsarkar, M.S., Mistry, A.R., Buckley, S.M., Kemball-Cook, G., Mosley, K.L., Mitrophanous, K., Radcliffe, P., Holder, M.V., Brittan, M., Georgiadis, A., Al-Allaf, F., Bigger, B.W., Gregory, L.G., Cook, H.T., Ali, R.R., Thrasher, A., Tuddenham, E.G., Themis, M. and Coutelle, C. (2004). Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood, 104:2714-21.
130. Wang, J., Wiley, J.M., Luddy, R., Greenberg, J., Feuerstein, M.A. and Bussel, J.B. (2005). Chronic immune thrombocytopenic purpura in children: assessment of rituximab treatment. J. Pediatr., 146: 217-21.
131. Wang, Z., Allen, J.M., Riddell, S.R., Gregorevic, P., Storb, R., Tapscott, S.J., Chamberlain, J.S. and Kuhr, C.S. (2007a). Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy. Hum. Gene Ther. 18: 18-26.
132. Wang, Z., Kuhr, C.S., Allen, J.M., Blankinship, M., Gregorevic, P., Chamberlain, J.S., Tapscott, S.J. and Storb, R. (2007b). Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol. Ther., 15: 1160-6.
133. Watanabe, T., Masuyama, J., Sohma, Y., Inazawa, H., Horie, K., Kojima, K., Uemura, Y., Aoki, Y., Kaga, S., Minota, S., Tanaka, T., Yamaguchi, Y., Kobayashi, T. and Serizawa, I. (2006). CD52 is a novel costimulatory molecule for induction of CD4+ regulatory T cells. Clin. Immunol., 120: 247-59.
134. Wong, W. and Wood, K.J. (2004). "Transplantation tolerance by donor MHC gene transfer. Curr. Gene Ther., 4: 329-36.
135. Wood, K.J. and Sakaguchi, S. (2003). Regulatory T cells in transplantation tolerance. Nat. Rev. Immunol., 3: 199-210.
136. Woodle, E.S., Xu, D., Zivin, R.A., Auger, J., Charette, J., O'Laughlin, R., Peace, D., Jollife, L.K., Haverty, T., Bluestone, J.A. and Thistlethwaite, J.R., Jr. (1999). Phase I trial of a humanized, Fc receptor nonbinding OKT3 antibody, huOKT3gamma1(Ala-Ala) in the treatment of acute renal allograft rejection. Transplantation, 68: 608-16.
137. Wu, H., Reding, M., Qian, J., Okita, D.K., Parker, E., Lollar, P., Hoyer, L.W. and Conti-Fine, B.M. (2001). Mechanism of the immune response to human factor VIII m murine hemophilia A. Thromb. Haemost., 85: 125-33.
138. Xu, L., Gao, C., Sands, M.S., Cai, S.R., Nichols, T.C., Bellinger, D.A., Raymer, R.A., McCorquodale, S. and Ponder, K.P. (2003). Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a
139. Yagi, H., Nomura, T., Nakamura, K., Yamazaki, S., Kitawaki, T., Hori, S., Maeda, M., Onodera, M., Uchiyama, T., Fujii, S. and Sakaguchi, S. (2004). Crucial role of FOXP3 in. the development and function of human CD25+CD4+ regulatory T cells. Int. Immunol., 16: 1643-56.
140. Ye, P., Thompson, A.R., Sarkar, R., Shen, Z., Lillicrap, D.P., Kaufman, R.J., Ochs, H.D., Rawlings, D.J. and Mao, C.H. (2004). Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol. Ther., 10: 117-26.
141. Yuasa, K., Yoshimura, M., Urasawa, N., Ohshima, S., Howell, J.M., Nakamura, A., Hijikata, T., Miyagoe-Suzuki, Y. and Takeda, S. (2007). Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products. Gene Ther., June 21: [Epub ahead of print].
142. Zecca, M., Nobili, B., Ramenghi, U., Perrotta, S., Amendola, G., Rosito, P., Jankovic, M., Pierani, P., De Stefano, P., Bonora, M.R. and Locatelli, F. (2003). Rituximab for the treatment of refractory autoimmune hemolytic anemia in children. Blood, 101: 3857-61.
143. Zhang, J., Xu, L., Haskins, M.E. and Parker Ponder, K. (2004). Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs. Blood, 103: 143-51.